20 Is The Loneliest Number: FDA Disease Meeting Schedule Leaves Some Worried About Exclusion

Conflict among groups with conditions both included and not included in the preliminary list of public meeting topics prompts FDA to reiterate that the diseases will be selected because better clinical measures are needed, not because the agency will prioritize them over others.

FDA is working to allay advocates’ fears that the 20 disease areas to be selected for public meetings will be the agency’s sole focus during the PDUFA V program on patient input.

The meetings are part of the prescription drug user fee program’s increased efforts to gain patient opinions about pending treatments. FDA agreed as part of the reauthorization to better incorporate patient opinions about risk tolerance and other drug development issues into its approval decisions.

To help reviewers better understand patient perspectives, FDA will conduct four public meetings per year during the five-year PDUFA cycle. Each will focus on a different disease area (Also see "Disease-Of-The-Quarter Club: FDA Plans 20 Patient Network Meetings Over Five Years" - Pink Sheet, 18 Jun, 2012.).

The agency developed a list of 39 candidate topics for the meetings, about one-third of them rare diseases, but it drew the ire of some patient groups.

Diane Dorman, vice president of public policy for the National Organization for Rare Disorders, said during a recent conference on the PDUFA reauthorization sponsored by the Drug Information Association and FDA that the list has pitted disease organizations against each other over the conditions on the list.

Some of that anger may come out during a public meeting on the preliminary list scheduled for Oct. 25.

Theresa Mullin, director of the Center for Drug Evaluation and Research’s Office of Planning and Informatics, said groups were worried FDA would focus on those diseases and neglect others. She said those fears were unexpected and are unjustified.

“What it is not is any sort of priority list for FDA,” Mullin, who helped negotiate PDUFA V on behalf of FDA, said during the conference. “There are a lot of very critical diseases that have unmet medical needs, are life-threatening, that are not on that list. Those diseases are also priorities for us.”

Each meeting will include discussions of the impact of the disease on patients, unmet needs and the quality of existing treatments.

The information is expected to help reviewers better understand patient needs and risk tolerance, as well as spur further research. Mullin said investigations of new patient-reported outcome measures could begin as a result of the comments received.

Mullin also said the proposed diseases were chosen in part because existing measures are inadequate.

“This is not to try to address all the important diseases, just ones where, in fact, we don’t have very good clinical measures, we don’t have good objective measures now, and we need to develop the best measures we can to encourage drug development,” she said.

Mullin mentioned chronic fatigue syndrome as an example because it does not have a definitive set of symptoms and obtaining more patient information would help develop more drugs for the treatment armamentarium.

FDA already has initiated a separate effort to work with patients and advocates to explore ways to develop more drugs for it and other conditions (Also see "Chronic Fatigue Syndrome Drug Development Efforts Get A Boost From FDA" - Pink Sheet, 13 Sep, 2012.).

Mullin said the agency will attempt to identify enough disease areas to schedule meetings for the first two years of PDUFA V, rather than compile the entire list. She was unsure how the next selections would be made for later meetings.

Will FDA Add More Diseases To The Meetings?

Given the advocacy community’s concerns about the list, FDA also could try to consolidate more conditions into the meetings. Dorman suggested some diseases could be grouped together, such as by body system, to include more conditions in the meetings.

But Mullin said that also could create problems, given the wide variety of perspectives that would be received.

Eric Gascho, director of government affairs for the National Health Council, said during the conference that FDA should remember some conditions have various states where patient perspectives could vary.

He also suggested that advocates be mindful that there will be many other opportunities for engagement outside the disease-specific meetings.

“You really do have to make sure that you’re hearing from a wide range of people, even [those] who have the same condition,” Gascho said.

Patient Group Consultation Meetings Have Begun

FDA also is convening several of what it is calling “consultation meetings” with patient stakeholders to talk about drug development issues and help formulate the direction of the disease-specific public meetings.

Problems to be discussed include: resolving conflicting opinions among stakeholders, balancing the priorities of different disease areas within FDA’s limited resources, and ensuring groups outside the Washington D.C. area gain equal access to the talks.

The bi-monthly discussions began Oct. 10 and are expected to continue throughout PDUFA V (Also see "FDA Adds “Consultation Meetings” To Patient-Focused Drug Development Effort" - Pink Sheet, 23 Sep, 2012.).

PDUFA V also includes specific provisions for increased expert consulting related to rare diseases to gain more input on orphan applications (Also see "PDUFA V: Accelerated Approval Expansion May Outshine Rare Disease Improvements" - Pink Sheet, 17 Sep, 2012.).

In addition, FDA has started training reviewers about the issues associated with orphan applications. The agency also is working with the National Institutes of Health to develop research investigator training for orphan drug development.